CML-paed II

Philadelphia chromosome-positive chronic myeloid leukaemia (CML)

Multicenter, non-randomized, open, prospective clinical trial

Primary objectives:
Assessment of antileukaemic activity of imatinib in children and adolescents with Philadelphia chromosome-positive chronic myeloid
leukaemia

Secondary objectives:
Assessment of the time-to event-efficacy variables
Correlation of the quality of haematological, cytogenetical and molecular remission in children and Adolescents with CML on ongoing imatinib therapy with survival
Safety of imatinib

Newly diagnosed patients with BCR-ABL-positive CML will be treated with imatinib. Serial monitoring of treatment response is performed in one month intervals during the first three months of treatment and in three months intervals thereafter. Patients with non-response, poor response (either molecular, cytogenetic, or hematologic non-/poor response) or progress of the disease while under imatinib treatment will stop imatinib and undergo stem cell transplantation. All responders to imatinib treatment with an HLA matched donor will undergo stem cell transplantation not later than 2 years after diagnosis.

• Newly diagnosed Philadelphia-chromosome positive or BCR-ABL-positive CML
• Written informed consent

• CML without BCR-ABL rearrangement detectable by PCR
• Pretreatment with Interferon-alpha or any other cytostatic drug with the exception of hydroxyurea or anagrelide (note: anagrelide is not approved n Germany for treatment of CML). However, these patients may be registered as observational patients.
• Any other severe underlying disease beside CML.
• Age > 18 years
• Pregnant or lactating women
• Subjects unlikely to comply with the requirements of the protocol